Patient-reported Outcomes and Burden of Disease in Paediatric Patients with Psoriasis: Real-world Data from EU5 and US.
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ABSTRACT:
SUBMITTER: Seyger MMB
PROVIDER: S-EPMC10128148 | biostudies-literature | 2023 Apr
REPOSITORIES: biostudies-literature
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Patient-reported Outcomes and Burden of Disease in Paediatric Patients with Psoriasis: Real-world Data from EU5 and US.
Seyger Marieke M B MMB Paller Amy A Sticherling Michael M Bachhuber Teresa T Thomas Nicolas N Hetherington James J Lucas James J Richardson Craig C Augustin Matthias M
Acta dermato-venereologica 20230419
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Project description:Data on the economic and humanistic burden of non-alcoholic steatohepatitis (NASH) are scarce. This study assessed the comparative burden of NASH, relative to a representative sample from the general population and a type 2 diabetes mellitus (T2DM) cohort, in terms of health-related quality of life, work productivity and activity impairment (WPAI), and healthcare resource use.MethodsData across 5 European countries came from the 2016 National Health and Wellness Survey, a nationally representative patient-reported outcomes survey. Outcomes included mental (MCS) and physical (PCS) component scores from the Short-Form (SF)-36v2, WPAI scores, self-reported physician diagnosis of sleep difficulties, anxiety, and depression, and healthcare resource use: healthcare professional visits, hospital visits, and emergency room visits in the previous 6 months. Bivariate and multivariable analyses were conducted for each outcome and comparative group.ResultsAfter adjusting for matching criteria and covariates, patients with NASH (n = 184) reported significantly worse health-related quality of life, worse WPAI scores, and more healthcare resource use than the general population (n = 736) (MCS 39.22 vs. 45.16, PCS 42.84 vs. 47.76; overall work impairment 49.15% vs. 30.77%, healthcare professional visits 10.73 vs. 6.01, emergency room visits 0.57 vs. 0.22, hospitalizations 0.47 vs. 0.17, p ≪0.05 for all). Patients with NASH did not differ from patients with T2DM (n = 368) on PCS and WPAI scores, suggesting a similar impairment on work and daily activities, but did report significantly worse mental status (MCS 39.64 vs. 43.64, p ≪0.05) and more healthcare resource use than those with T2DM (healthcare professional visits 10.85 vs. 7.86, emergency room visits 0.65 vs. 0.23, hospitalizations 0.39 vs. 0.19, p ≪0.05 for all).ConclusionsThese findings suggest that the burden of NASH may be underestimated, highlighting the unmet needs of patients with NASH.Lay summaryThese findings show that patients with non-alcoholic steatohepatitis (NASH) experience a significant burden of illness, in terms of health-related quality of life, work productivity and activity impairment, and healthcare resource use. As there is currently no approved treatment for NASH, these findings highlight the unmet medical need of patients with NASH.
| S-EPMC7001541 | biostudies-literature
Project description:Background:Advanced breast cancer (abc) represents a substantial burden for patients and caregivers. In the present study, we aimed to estimate quality of life (qol), utility, productivity loss, pain, health care resource utilization, and costs for patients with abc, and qol, utility, and productivity loss for their caregivers. Methods:This multicentre prospective non-interventional study was conducted in Canada. Eligible participants were postmenopausal women with estrogen receptor-positive, her2-negative unresectable abc and their caregivers. Validated questionnaires were used to measure qol, utility, productivity loss, and pain. Patients and caregivers were classified into 4 health states typically used in oncology economic modelling: first-line progression-free (1l-pf), first-line progressive disease (1l-pd), second- or subsequent-line progression-free (≥2l-pf), and second- or subsequent-line progressive disease (≥2l-pd). Results:Most patients and caregivers accepted to participate, with total recruitment of 202 patients and 78 caregivers. Compared with patients in pf, patients in pd had lower mean qol scores (52.9 ± 29.9 for 1l-pd vs. 68.2 ± 23.2 for 1l-pf, and 54.0 ± 23.6 for ≥2l-pd vs. 66.0 ± 22.1 for ≥2l-pf), lower mean utility values (0.64 ± 0.22 for 1l-pd vs. 0.73 ± 0.20 for 1l-pf, and 0.65 ± 0.25 for ≥2l-pd vs. 0.74 ± 0.18 for ≥2l-pf), and greater productivity loss (39.4 ± 27.7 for 1l-pd vs. 27.5 ± 30.1 for 1l-pf, and 37.6 ± 29.2 for ≥2l-pd vs. 32.0 ± 29.0 for ≥2l-pf). Compared with caregivers of patients in pf, caregivers of patients in pd had lower qol scores and utility values, and greater productivity loss. Conclusions:Study results indicate that, for patients and caregivers, pd health states are associated with a deterioration of qol and utility and a decrease in productivity in both 1l and ≥2l.
| S-EPMC6092048 | biostudies-literature
Project description:BackgroundHidradenitis suppurativa (HS) is a chronic, inflammatory, debilitating skin disease characterized by painful deep lesions and associated with substantial disease burden.ObjectivesThe objective of this study was to describe physician- and patient-reported clinical unmet needs from a real-world perspective.MethodsThis study used data from the Adelphi HS Disease Specific Programme, a point-in-time survey of dermatologists and their patients with HS in Europe and the United States. Dermatologists completed patient record forms (PRFs) for 5-7 consecutively consulting patients with HS; patients or carers of patients also optionally completed a patient/carer self-completion questionnaire (PSC/CSC). Data collection included demographics, symptomatology and impact on quality of life (QoL).ResultsDermatologists (N = 312) completed PRFs for 1787 patients with HS; patient- and carer-reported questionnaires (PSC/CSC) were completed for 33.1% (591/1787) of patients. The mean age was 34.4 ± 12.2 years and 57.6% of patients were female (1029/1787). Physician-judged disease severity at sampling was categorized as mild in 66.0% (1179/1787), moderate in 29.3% (523/1787) and severe in 4.7% (85/1787) of patients. Deterioration or unstable condition over the previous 12 months was described by 17.1% [235/1372] and 12.6% [41/325] of physician- and patient/carer-reported cases, respectively. Despite receiving treatment, high proportions of patients still experienced symptoms at sampling (general pain/discomfort [49.5%, 885/1787]; inflammation/redness of lesions/abscesses [46.1%, 823/1787] and itching [29.9%, 535/1787]); these symptoms were more frequent in patients with moderate or severe disease. Patients reported a mean Dermatology Life Quality Index score of 5.9 ± 5.4 (555/591; mild, 4.1 ± 4.3; moderate, 9.4 ± 5.4; severe, 13.3 ± 5.5) and a mean Hidradenitis Suppurativa Quality of Life score of 11.0 ± 10.6 (518/591; mild, 7.6 ± 8.3; moderate, 17.7 ± 10.0; severe, 31.0 ± 15.4) indicating a substantial impact on QoL.ConclusionsPatients with HS experienced a high disease burden despite being actively treated by a dermatologist. This study demonstrates that the burden of HS disease is generally poorly managed with a considerable impact observed on patients' QoL.
| S-EPMC9545202 | biostudies-literature
Project description:BackgroundEosinophilic esophagitis is a chronic inflammatory disorder of the esophagus. This real-world study used patient and physician surveys to describe the clinical characteristics and disease burden of eosinophilic esophagitis-overall and in a subgroup of patients with dysphagia despite treatment.MethodsData analyzed in this study were collected in 2020 from US and EU patients with eosinophilic esophagitis. Eligible patients were aged ≥ 12 years with a diagnosis of eosinophilic esophagitis, had an esophageal count of ≥ 15 eosinophils/high-power field at diagnosis, and were currently prescribed treatment for eosinophilic esophagitis.ResultsOverall, 1001 patients were included, of whom 356 (36%) had dysphagia despite treatment. Demographics and clinical characteristics were similar in both populations. The severity of eosinophilic esophagitis was mild in more patients overall (69%) versus those with dysphagia despite treatment (48%). Patient disease history was similar in both populations, with some exceptions: common patient-reported symptoms were dysphagia (70% and 86%) and heartburn/acid reflux (55% and 49%), and common physician-reported symptoms were dysphagia (75% and 91%) and food impaction (46% and 52%). Treatment history was similar in both populations; overall, the most common treatments were proton pump inhibitors (83%) and topical corticosteroids (51%). Patients reported slightly more days with symptoms, higher impacts on activities of daily living, and slightly higher anxiety or depression in the dysphagia-despite-treatment population versus the overall population.ConclusionsEosinophilic esophagitis presents severe symptoms and comorbidities that substantially impact patients' well-being and quality of life. Greater awareness of and novel treatments for eosinophilic esophagitis are needed.
| S-EPMC11297626 | biostudies-literature
Project description:BackgroundThe current analysis utilized data collected via an online patient community platform, PatientsLikeMe (PLM) to compare patient-reported experiences in patients with major depressive disorder (MDD) with suicidal ideation (MDSI) to those with MDD but without suicidal ideation.MethodsPLM members who joined PLM between May-2007 and February-2018 and reported a diagnosis of MDD were included. The MDSI cohort included patients with MDD who reported at least one suicide-related symptom at a severity greater than "none". Demographics, comorbidities, symptoms, and side-effects were compared between MDSI and MDD cohorts. Factors correlated with suicidal ideation (SI) were determined by a random forest procedure.ResultsPatients in the MDSI cohort (n = 266) were younger (median age, 36 vs 44 years) with an earlier disease onset (before 30 years, 83% vs 71%), and a longer diagnosis latency (median, 4 vs 2 years) vs patients in the MDD cohort (n = 11,963). Majority of patients were women in both cohorts (73% vs 83%). Median number of psychiatric comorbidities was higher in the MDSI cohort (4 vs 3). Unprompted symptoms (e.g., loneliness, feeling of hopelessness, social anxiety, impulsivity, and self-hating thoughts) were more frequent in the MDSI cohort. Hopelessness, loneliness, anhedonia, social anxiety, and younger age were highly correlated with suicidal ideation.ConclusionsThis analysis utilized patient-reported data to better understand symptoms, experiences, and characteristics of patients with MDSI compared to patients with MDD. The results identified various risk factors correlated with suicidal ideation that may help guide clinical judgement for patients with MDD who may not voluntarily report suicidal ideation.
| S-EPMC7376651 | biostudies-literature
Project description:Background: Generalized pustular psoriasis (GPP) is a rare, systemic disease characterized by persistent or recurrent flares of painful neutrophilic pustules. There is limited real-world evidence characterizing patients with GPP. Objectives: To establish the distinguishing characteristics of GPP relative to plaque psoriasis, and help inform future treatment decisions and improve patient outcomes. Methods: North American adults with GPP or plaque psoriasis (without pustules) identified from CorEvitas' Psoriasis Registry were included in this dataset. Registry enrollment data, including patient sociodemographics, disease characteristics, medication use, and patient-reported outcome measures were compared for patients with GPP vs those with plaque psoriasis. This study was descriptive, and no hypothesis tests were performed. Results: In this sample, patients with GPP (N = 60) reported greater median (interquartile range) pain (20 [3-62] vs 5 [0-35]), fatigue (44 [15-73] vs 20 [4-50]), and itch (59 [10-85] vs 22 [5-70]) than those with plaque psoriasis (N = 4894). Descriptively, patients with GPP also reported more anxiety and depression (EQ-5D-3L: 38% vs 26%) and had more treatment experience (≥2 previous systemics: 15% vs 7%). Conclusions: A greater degree of symptom severity and impact on quality of life was reported by patients with GPP compared with plaque psoriasis in this sample. Importantly, patients with GPP had more treatment experience, suggesting that current treatment options do not adequately resolve the disease-highlighting the need to develop more effective GPP treatments.
| S-EPMC11361522 | biostudies-literature
Project description:ObjectivesTo assess tolerability of COVID-19 vaccination in patients with RA and controls based on patient-reported outcomes (PROs).MethodsIn total, 266 study participants were included at 6 ± 1 weeks after their second vaccination (BioNTech/Pfizer (72.2%), AstraZeneca (18.8%) and Moderna (9.0%)). In a cross-sectional, observational study design, PRO data were recorded regarding both total and symptom-level tolerability.ResultsOverall tolerability was very high according to the patients' self-assessment scores (1.71 for the first and 1.72 for the second vaccination, 6-point Likert scale [1 (very good) to 6 (very poor)]) and did not differ significantly between patients with RA (n = 204) and controls (n = 62). Self-rated overall tolerability regarding first vaccination was significantly better (P = 0.002) in patients receiving mRNA vaccines (n = 193, mean tolerability 1.59) as compared with vector-vaccinated patients (n = 73, mean tolerability 2.04). Homologous or heterologous vaccination regimens had no statistically significant effect on vaccine tolerability (P = 0.131). Reservations about the vaccination were rare (6.4% for the first and 6.0% for the second vaccination) but significantly associated with poorer overall tolerability (P < 0.001) and significantly reduced willingness to recommend vaccination to others (P < 0.001 for the first and P = 0.004 for the second vaccination).ConclusionBased on these real-world data, tolerability of COVID-19 vaccination was very good in both RA patients and controls. Reservations against COVID-19 vaccination were rare overall, but if present, associated with a significantly worse tolerability and a significantly lower degree of recommendation.
| S-EPMC11398971 | biostudies-literature
Project description:BackgroundIn breast cancer patients body image (BI) is a crucial aspect of quality of life (QoL). This study examined the postoperative impact of different surgical approaches on long-term BI analyzing real-world data to guide pre- and postoperative patient care and preserve QoL.MethodsEORTC QLQ-BR23 BI scores were collected electronically in 325 breast cancer patients within routine clinical care for a duration of 41.5 months (11/17/2016 - 4/30/2020) at predefined time points preoperatively and repeatedly up to two years after breast-conserving surgery (BCS) (n = 212), mastectomy alone (M) (n = 27) or mastectomy with immediate breast reconstruction (MIBR) (n = 86). Higher scores indicated better BI. A linear mixed regression model was used to analyze the impact of BCS, M and MIBR, as well as non-surgical therapies on BI at treatment initiation and over time.ResultsBI scores deteriorated by 5 points (95%-confidence interval (CI) -8.94 to -1.57, p≈0.005) immediately after BCS, by 7 points (95%-CI -12.13 to -1.80, p≈0.008) after MIBR and by 19 points (95%-CI -27.34 to -10.34, p < 0.001) after M. The change over time after BCS (+ 0.10 points per week, 95%-CI -0.17 to 0.38), MIBR (-0.07 points per week, 95%-CI -0.35 to 0.20) and M (+ 0.14 points per week, 95%-CI -0.19 to 0.48) were not statistically significant (each p > 0.05). At treatment initiation chemotherapy was associated with a 22-point decline (95%-CI -25.39 to -17.87, p < 0.001) in BI score, while radiotherapy was associated with a 5-point increase (95%-CI 1.74 to 9.02, p≈0.004). However, over time chemotherapy was associated with a score recovery (+ 0.28 points per week, 95%-CI 0.19 to 0.37, p < 0.001), whereas for radiotherapy a trend towards BI deterioration was observed (-0.11 points per week, 95%-CI -0.23 to 0.02, p≈0.101).ConclusionsBreast cancer surgery negatively affects BI. BCS and MIBR presumably harm BI less than M in the early postoperative period. Our data suggests BI to be deteriorating in the long term after MIBR while improving after BCS or M. Radiotherapy seems to have an additional negative long-term impact on BI. These findings should be confirmed in further studies to enable evidence-based patient information as part of preoperative shared decision-making and postoperative patient care.
| S-EPMC10375629 | biostudies-literature
Project description:ObjectivesThis analysis examined the association between psoriasis severity, assessed by body surface area (BSA) and the Investigator's Global Assessment (IGA; previously used only in clinical trials), and patient-reported outcomes (PROs) in a real-world setting.DesignCross-sectional analysis within the Corrona Psoriasis Registry, an independent, prospective registry.Setting70 dermatology practices in the USA.Participants1529 adult patients with psoriasis being treated with biological or non-biological systemic psoriasis treatment by 31 May 2016.Primary and secondary outcome measuresPsoriasis severity was assessed by percentage of affected BSA (mild (0%-5%), moderate (>5%-10%), severe (>10%-15%), very severe (>15%)) and IGA scores (clear/almost clear (0-1), mild (2), moderate (3), severe (4)). PROs (pain, itch, fatigue; Dermatology Life Quality Index [DLQI]; EuroQoL Visual Analogue Scale [EQ-VAS]; Work Productivity and Activity Impairment [WPAI]) were compared across BSA and IGA levels using analysis of variance and X2 tests. The association between psoriasis severity and PROs was examined using multivariable regression models.ResultsThe mean age was 50.6 years and 47% of patients were female. Consistently with more severe psoriasis, symptoms worsened, DLQI scores increased (p<0.05 for each level of BSA and IGA), EQ-VAS decreased (p<0.05 for each level of BSA and IGA) and WPAI scores increased. By BSA score, moderate to very severe psoriasis was associated with poorer outcomes for the 'impairment while working' and 'daily activities impaired' WPAI domains (all p<0.05 vs mild psoriasis). Very severe psoriasis was associated with increased 'work hours missed' and 'work hours affected' (both p<0.05 vs mild psoriasis) Findings were similar by IGA. Results were confirmed by multivariable regression analyses.ConclusionsIn a real-world setting, more severe psoriasis, assessed by BSA and IGA, was consistently associated with worse PROs.
| S-EPMC6500315 | biostudies-literature
Project description:Background: The t:slim X2™ insulin pump with Control-IQ™ technology, an advanced hybrid closed-loop system, became available in the United States in early 2020. Real-world outcomes with use of this system have not yet been comprehensively reported. Methods: Individuals with type 1 diabetes (T1D) (≥14 years of age) who had ≥21 days of pump usage data were invited via email to participate. Participants completed psychosocial questionnaires (Technology Acceptance Scale [TAS], well-being index [WHO-5], and Diabetes Impact and Devices Satisfaction [DIDS] scale) at timepoint 1 (T1) (at least 3 weeks after starting Control-IQ technology) and the DIDS and WHO-5 at timepoint 2 (T2) (4 weeks from T1). Patient-reported outcomes (PROs) and glycemic outcomes were reviewed at each timepoint. Results: Overall, 9,085 potentially eligible individuals received the study invite. Of these, 3,116 consented and subsequently 1,435 participants completed questionnaires at both T1 and T2 and had corresponding glycemic data available on the t:connect® web application. Time in range was 78.2% (70.2%-85.1%) at T1 and 79.2% (70.3%-86.2%) at T2. PROs reflected high device-related satisfaction and reduced diabetes impact at T2. Factors contributing to high trust in the system included sensor accuracy, improved diabetes control, reduction in extreme blood glucose levels, and improved sleep quality. In addition, participants reported improved quality of life, ease of use, and efficient connectivity to the continuous glucose monitoring system as being valuable features of the system. Conclusions: Continued real-world use of the t:slim X2 pump with Control-IQ technology showed improvements in psychosocial outcomes and persistent achievement of recommended TIR glycemic outcomes in people with T1D.
| S-EPMC7868573 | biostudies-literature